Drug Discovery and Development Notes

Definition: The process of discovering biological molecules (typically proteins) that can be targeted by drugs.
Steps:
1. Disease Understanding: Analyze disease mechanisms to identify potential targets.
2. Biomarker Discovery: Identify biomarkers that indicate disease presence or progression.
3. High-Throughput Screening: Utilize technology to screen large libraries of compounds against targets.
Tools: Genomics, proteomics, and bioinformatics are commonly used.

Purpose: To evaluate the safety and efficacy of drug candidates before human trials.
Phases:
1. In Vitro Testing: Conduct tests using cell cultures to assess biological activity.
2. Animal Studies: Evaluate pharmacokinetics (ADME - Absorption, Distribution, Metabolism, Excretion) and toxicology in animal models.
Outcomes: Data collected will guide the decision to move to clinical trials.

Overview: Research studies conducted on humans to assess the safety and efficacy of new drugs.
Phases:
1. Phase I: Tests safety, dosage, and side effects in a small group (20-100 participants).
2. Phase II: Evaluates efficacy and side effects in a larger group (100-300 participants).
3. Phase III: Confirms effectiveness, monitors side effects, and compares with standard treatments in a large population (1,000-3,000 participants).
4. Phase IV: Post-marketing surveillance to assess long-term effects and further evaluate risks.
Regulatory submission occurs after successful Phase III results.

Role: Ensure compliance with regulations and laws governing drug development.
Key Agencies:
- FDA (Food and Drug Administration) in the USA
- EMA (European Medicines Agency) in Europe
Functions:
- Review and approval of clinical trial applications (CTAs).
- Submission of New Drug Applications (NDAs) or Biologics License Applications (BLAs).
- Post-approval monitoring and reporting of adverse events.

Definition: The process of combining different chemical substances to create a final medicinal product.
Considerations:
- Stability: Ensuring the drug maintains efficacy over time.
- Bioavailability: Designing the formulation to ensure optimal absorption in the body.
- Delivery Method: Choosing between oral, injectable, topical, etc., depending on the drug characteristics and target condition.
Development involves:
- Pre-formulation studies to assess physical and chemical properties.
- Formulation optimization to enhance performance and patient compliance.

Involves discovering biological molecules (often proteins) that drugs can target.
Steps include understanding disease mechanisms, discovering biomarkers indicating disease, and utilizing high-throughput screening for compound testing.
Employs tools like genomics, proteomics, and bioinformatics to streamline the process.

Aims to assess safety and efficacy of drug candidates prior to human testing.
Comprises in vitro testing with cell cultures for biological activity evaluation and animal studies for pharmacokinetics (Absorption, Distribution, Metabolism, Excretion) and toxicology assessments.
Collected data informs decisions on advancing to clinical trials.

Research studies designed to evaluate new drugs on human subjects for safety and efficacy.
Phases include:
- Phase I: Tests safety and dosages in a small cohort (20-100).
- Phase II: Assesses efficacy in a larger group (100-300).
- Phase III: Confirms effectiveness and monitors side effects in a large demographic (1,000-3,000).
- Phase IV: Conducts post-marketing surveillance for long-term effects and risks.
Regulatory submission follows successful Phase III trials.

Focuses on compliance with drug development regulations and laws.
Key regulatory bodies include the FDA in the USA and EMA in Europe.
Responsibilities include reviewing clinical trial applications (CTAs), submitting New Drug Applications (NDAs) or Biologics License Applications (BLAs), and monitoring post-approval adverse events.

Entails combining various chemical substances to create the final medicinal product.
Key considerations involve:
- Stability to maintain drug efficacy over time.
- Bioavailability to optimize drug absorption.
- Selection of delivery methods like oral, injectable, or topical based on drug and condition.
Development includes pre-formulation studies for physical/chemical property assessment and optimization for enhanced performance and patient adherence.